Familial Amyloid Neuropathies Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

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Familial Amyloid Neuropathies Pipeline Drugs Market Report Overview

Familial amyloid neuropathy is a slowly progressive condition characterized by the buildup of abnormal deposits of a protein called amyloid (amyloidosis) in the body’s organs and tissues. Symptoms include numbness, tingling, pins, and needles in the feet and hands, weakness and pain in the arms and legs, loss of sensation, urinary retention, reduced sweating, ankle swelling, fatigue, nausea, weight loss, dizziness, and fainting.

The Familial Amyloid Neuropathies pipeline drugs market research report provides comprehensive information on the therapeutics under development for Familial Amyloid Neuropathies, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history, and the latest news and press releases. Additionally, the report offers an overview of key players involved in therapeutic development for Familial Amyloid Neuropathies and features dormant and discontinued projects.

Familial Amyloid Neuropathies Pipeline Drugs Market Segmentation by Targets

Some of the key targets of the Familial Amyloid Neuropathies pipeline drugs market are Transthyretin, Alpha Synuclein, Amyloid Beta A4 Protein, and Microtubule Associated Protein Tau. Transthyretin leads this segment of the Familial Amyloid Neuropathies pipeline drugs market.

Familial Amyloid Neuropathies Pipeline Drugs Market Analysis, by Targets

Familial Amyloid Neuropathies Pipeline Drugs Market Analysis, by TargetsFor more target insights on Familial Amyloid Neuropathies pipeline drugs market, download a free report sample

Familial Amyloid Neuropathies Pipeline Drugs Market Segmentation by Mechanisms of Action

Some of the key mechanisms of action of the Familial Amyloid Neuropathies pipeline drugs market are Transthyretin Inhibitor, Alpha Synuclein Inhibitor, Amyloid Beta A4 Protein Inhibitor, and Microtubule Associated Protein Tau Inhibitor. Transthyretin Inhibitor leads the Familial Amyloid Neuropathies pipeline drugs market in terms of MoA.

Familial Amyloid Neuropathies Pipeline Drugs Market Analysis, by Mechanisms of Action

Familial Amyloid Neuropathies Pipeline Drugs Market Analysis, by Mechanisms of ActionFor more MoA insights into the Familial Amyloid Neuropathies pipeline drugs market, download a free report sample

Familial Amyloid Neuropathies Pipeline Drugs Market Segmentation by Routes of Administration

The key routes of administration in the Familial Amyloid Neuropathies pipeline drugs market are intravenous, subcutaneous, parenteral, and oral. Intravenous leads the Familial Amyloid Neuropathies pipeline drugs market in terms of RoA. 

Familial Amyloid Neuropathies Pipeline Drugs Market Analysis, by Routes of Administration

Familial Amyloid Neuropathies Pipeline Drugs Market Analysis, by Routes of AdministrationFor more RoA insights into the Familial Amyloid Neuropathies pipeline drugs market, download a free report sample

Familial Amyloid Neuropathies Pipeline Drugs Market Segmentation by Molecule Types

The molecule types in the Familial Amyloid Neuropathies pipeline drugs market are small molecule, antisense oligonucleotide, antisense RNAi oligonucleotide, gene therapy, antibody, fusion protein, and monoclonal antibody. Small molecule leads the Familial Amyloid Neuropathies pipeline drugs market in terms of molecule types.

Familial Amyloid Neuropathies Pipeline Drugs Market Analysis, by Molecule Types

Familial Amyloid Neuropathies Pipeline Drugs Market Analysis, by Molecule TypesFor more molecule-type insights into the Familial Amyloid Neuropathies pipeline drugs market, download a free report sample

Familial Amyloid Neuropathies Pipeline Drugs Market - Competitive Landscape

Some of the leading companies in the Familial Amyloid Neuropathies pipeline drugs market are Akcea Therapeutics Inc, Alnylam Pharmaceuticals Inc, Regeneron Pharmaceuticals Inc, ADRx Inc, BSIM Therapeutics SA, Corino Therapeutics Inc, Novo Nordisk AS, and Proclara Biosciences Inc. Akcea Therapeutics Inc, Alnylam Pharmaceuticals Inc, and Regeneron Pharmaceuticals Inc have the highest number of Familial Amyloid Neuropathies drugs under development in 2022.

Familial Amyloid Neuropathies Pipeline Drugs Market Analysis, by Companies

Familial Amyloid Neuropathies Pipeline Drugs Market Analysis, by CompaniesTo know more about the leading Familial Amyloid Neuropathies pipeline drugs market players, download a free report sample

Familial Amyloid Neuropathies Pipeline Drugs Market Overview

Key Targets Transthyretin, Alpha Synuclein, Amyloid Beta A4 Protein, and Microtubule Associated Protein Tau
Key Mechanisms of Action Transthyretin Inhibitor, Alpha Synuclein Inhibitor, Amyloid Beta A4 Protein Inhibitor, and Microtubule Associated Protein Tau Inhibitor
Key Routes of Administration Intravenous, Subcutaneous, Parenteral, and Oral
Key Molecule Types Small Molecule, Antisense Oligonucleotide, Antisense RNAi Oligonucleotide, Gene Therapy, Antibody, Fusion Protein, and Monoclonal Antibody
Leading Companies Akcea Therapeutics Inc, Alnylam Pharmaceuticals Inc, Regeneron Pharmaceuticals Inc, ADRx Inc, BSIM Therapeutics SA, Corino Therapeutics Inc, Novo Nordisk AS, and Proclara Biosciences Inc

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Familial Amyloid Neuropathies.
  • The pipeline guide reviews pipeline therapeutics for Familial Amyloid Neuropathies by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration to discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Familial Amyloid Neuropathies therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Familial Amyloid Neuropathies therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA), and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews the latest news related to pipeline therapeutics for Familial Amyloid Neuropathies

Reasons to Buy

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with a potentially strong product portfolio and create effective counterstrategies to gain a competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Familial Amyloid Neuropathies.
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and their most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding the Familial Amyloid Neuropathies pipeline depth and focus of Indication therapeutics.
  • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understanding from the know-how what drove them from the pipeline.

ADRx Inc
Akcea Therapeutics Inc
Alnylam Pharmaceuticals Inc
BSIM Therapeutics SA
Corino Therapeutics Inc
Novo Nordisk AS
Proclara Biosciences Inc
Regeneron Pharmaceuticals Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Familial Amyloid Neuropathies – Overview

Familial Amyloid Neuropathies – Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Familial Amyloid Neuropathies – Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Familial Amyloid Neuropathies – Companies Involved in Therapeutics Development

ADRx Inc

Akcea Therapeutics Inc

Alnylam Pharmaceuticals Inc

BSIM Therapeutics SA

Corino Therapeutics Inc

Novo Nordisk AS

Proclara Biosciences Inc

Regeneron Pharmaceuticals Inc

Familial Amyloid Neuropathies – Drug Profiles

Antibody to Inhibit TTR for Familial Transthyretin Amyloidosis – Drug Profile

Product Description

Mechanism Of Action

CLR-01 – Drug Profile

Product Description

Mechanism Of Action

doxycycline hyclate – Drug Profile

Product Description

Mechanism Of Action

eplontersen sodium – Drug Profile

Product Description

Mechanism Of Action

NNC-6019 – Drug Profile

Product Description

Mechanism Of Action

NPT-189 – Drug Profile

Product Description

Mechanism Of Action

NTLA-2001 – Drug Profile

Product Description

Mechanism Of Action

patisiran – Drug Profile

Product Description

Mechanism Of Action

Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy – Drug Profile

Product Description

Mechanism Of Action

tolcapone – Drug Profile

Product Description

Mechanism Of Action

vutrisiran – Drug Profile

Product Description

Mechanism Of Action

Familial Amyloid Neuropathies – Dormant Projects

Familial Amyloid Neuropathies – Discontinued Products

Familial Amyloid Neuropathies – Product Development Milestones

Featured News & Press Releases

Sep 07, 2022: Ionis presents positive results from phase 3 NEURO-TTRansform study at International Symposium on Amyloidosis

Jun 22, 2022: AstraZeneca-Ionis’ eplontersen meets endpoints in Phase III ATTRv-PN trial

Oct 27, 2021: Alnylam reports positive topline 18-month results from HELIOS-A phase 3 study of Vutrisiran in patients with hATTR amyloidosis with polyneuropathy

Jun 24, 2021: Alnylam announces U.S. Food and Drug Administration acceptance of new drug application for investigational Vutrisiran for the treatment of the polyneuropathy of hereditary ATTR amyloidosis

Nov 13, 2017: European Medicines Agency (EMA) Grants Alnylam Accelerated Assessment of Patisiran for Patients with Hereditary ATTR (hATTR) Amyloidosis

Nov 02, 2017: Sanofi and Alnylam Present Positive Complete Results from APOLLO Phase 3 study of Investigational Patisiran in Hereditary ATTR (hATTR) Amyloidosis Patients with Polyneuropathy

Sep 20, 2017: Arbutus’ LNP Licensee Alnylam Announces Positive Phase 3 Results for LNP-Enabled Patisiran Program

Sep 20, 2017: Sanofi and Alnylam Report Positive Topline Results from APOLLO Phase 3 Study of Patisiran in Hereditary ATTR (hATTR) Amyloidosis Patients with Polyneuropathy

Apr 26, 2017: Alnylam Reports Final 24-Month Results from Phase 2 Open-Label Extension Study of Patisiran, an Investigational RNAi Therapeutic in Development for the Treatment of Hereditary ATTR (hATTR) Amyloidosis

Oct 10, 2016: Data Monitoring Committee Recommends Continuation of APOLLO Phase 3 Clinical Trial of Patisiran for Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN)

Aug 16, 2016: BSIM Therapeutics was awarded research and development contract fund

Jul 01, 2016: Alnylam Reports New Results on patisiran at the XV International Symposium on Amyloidosis

Jun 27, 2016: Alnylam to Report New Patisiran Results at the XV International Symposium on Amyloidosis

Apr 20, 2016: Alnylam Reports Complete 18-Month Data from Ongoing Phase 2 Open-Label Extension Study of Patisiran, an Investigational RNAi Therapeutic Targeting Transthyretin for the Treatment of Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN)

Feb 01, 2016: Alnylam Completes Enrollment in APOLLO Phase 3 Study with Patisiran, an Investigational RNAi Therapeutic for Patients with Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

Table

List of Tables

Number of Products under Development for Familial Amyloid Neuropathies, 2022

Number of Products under Development by Companies, 2022

Number of Products under Development by Universities/Institutes, 2022

Products under Development by Companies, 2022

Products under Development by Universities/Institutes, 2022

Number of Products by Stage and Target, 2022

Number of Products by Stage and Mechanism of Action, 2022

Number of Products by Stage and Route of Administration, 2022

Number of Products by Stage and Molecule Type, 2022

Familial Amyloid Neuropathies – Pipeline by ADRx Inc, 2022

Familial Amyloid Neuropathies – Pipeline by Akcea Therapeutics Inc, 2022

Familial Amyloid Neuropathies – Pipeline by Alnylam Pharmaceuticals Inc, 2022

Familial Amyloid Neuropathies – Pipeline by BSIM Therapeutics SA, 2022

Familial Amyloid Neuropathies – Pipeline by Corino Therapeutics Inc, 2022

Familial Amyloid Neuropathies – Pipeline by Novo Nordisk AS, 2022

Familial Amyloid Neuropathies – Pipeline by Proclara Biosciences Inc, 2022

Familial Amyloid Neuropathies – Pipeline by Regeneron Pharmaceuticals Inc, 2022

Familial Amyloid Neuropathies – Dormant Projects, 2022

Familial Amyloid Neuropathies – Discontinued Products, 2022

Figures

List of Figures

Number of Products under Development for Familial Amyloid Neuropathies, 2022

Number of Products under Development by Companies, 2022

Number of Products under Development by Universities/Institutes, 2022

Number of Products by Top 10 Targets, 2022

Number of Products by Stage and Top 10 Targets, 2022

Number of Products by Top 10 Mechanism of Actions, 2022

Number of Products by Stage and Top 10 Mechanism of Actions, 2022

Number of Products by Top 10 Routes of Administration, 2022

Number of Products by Stage and Top 10 Routes of Administration, 2022

Number of Products by Top 10 Molecule Types, 2022

Number of Products by Stage and Top 10 Molecule Types, 2022

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